AMT-260

May 05, 2026 — uniQure N.V. (NASDAQ: QURE) reported first-quarter 2026 financial results and provided pipeline and regulatory updates. For AMT-130 (Huntington’s disease), uniQure said the FDA (Type A meeting minutes after an Oct 2025 pre-BLA discussion) would not agree that Phase I/II data versus an external control are sufficient as primary evidence of effectiveness for a marketing application; a Type B FDA meeting is scheduled in Q2 2026 to discuss a new trial design and statistical analysis plan for four-year data expected in Q3 2026. In the UK, following an MHRA pre-submission meeting, uniQure plans to submit a Marketing Authorization Application in Q3 2026 based on three-year data.

Pipeline: AMT-260 (refractory mesial temporal lobe epilepsy) enrollment is ongoing in cohort 2; cohort 1 (n=6) data with up to six months follow-up will be presented June 18–19, 2026. AMT-191 (Fabry disease) Phase I/II updates showed durable, dose-dependent α-Gal A activity increases and stable lyso-Gb3; all 11 dosed patients discontinued ERT, but mid/high-dose dosing is paused due to asymptomatic Grade 3 liver enzyme elevations (DLTs). AMT-162 (SOD1-ALS) development was discontinued.

Business/financial: HEMGENIX supply commitments to CSL Behring will end after mid-2026 batch deliveries; royalties/milestones unchanged. Cash/cash equivalents/current investments were $586.6M (Mar 31, 2026), with runway into 2H 2029; Q1 revenue $3.6M; net loss $53.5M ($0.85/share).